September 3, 2015
A new study from NEJM (Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia, N Engl J Med 2015; 373:920-928 September 3, 2015) reports that all 18 ET patients in a Phase 2 trial treated with the drug imetelstat exhibited decreased platelet levels, and 16 showed normalized blood cell counts. Eight patients participated at City of Hope and the rest were treated at hospitals in Germany and Switzerland. Most participants experienced only transient mild or moderate adverse affects. City of Hope physician David Snyder, M.D, associate chair and professor in City of Hope’s Department of Hematology & Hematopoietic Cell Transplantation, was senior author of the paper.
“Our trial was a first look at what happens when you treat ET patients with a drug that has a totally novel mechanism of action,” said Snyder, a co-principal investigator on the trial. “These studies open the door to test imetelstat effects in myelofibrosis, an MPN whose prognosis is much worse than ET.”
This represents the first data from a Phase 2 study on a new mechanism of action against the myeloproliferative neoplasms.